Chemogenetic Gene Therapy for Parkinson's Disease Using AAV Virus and Stereotactic Surgery
Summary
NIH registered a Phase 1 clinical trial (NCT07533591) evaluating a chemogenetic gene therapy called STP-001 for Parkinson's disease. The therapy uses an AAV viral vector with hM4Di chemogenetic effector delivered via stereotactic surgery to the subthalamic nucleus, combined with clozapine to modulate neuronal activity. The study aims to improve core motor symptoms in PD patients.
What changed
NIH added a clinical trial registration for a Phase 1 study of a novel chemogenetic gene therapy approach for Parkinson's disease. The trial will evaluate STP-001, an AAV viral vector carrying hM4Di chemogenetic effector, injected bilaterally into the subthalamic nucleus via stereotactic surgery, with clozapine activation.
Healthcare providers and clinical investigators in neurology and gene therapy should note this early-phase trial for Parkinson's disease research. The study represents an experimental approach combining gene therapy with chemogenetics for motor symptom intervention. Patients considering trial participation should consult the clinical investigators directly.
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Apr 17, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
Clinical Study of Chemogenetic Gene Therapy With AAV Virus for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus
Early Phase 1 NCT07533591 Kind: EARLY_PHASE1 Apr 16, 2026
Abstract
The investigators propose a gene therapy strategy for Parkinson's disease - a chemogenetic inhibition technique to intervene in the abnormal activity of the subthalamic nucleus in Parkinson's patients. The investigators design and construct a therapeutic injection agent called STP-001, through an efficient adeno-associated virus capsid (AAV), a neuronal promoter (hSyn), and a chemogenetic effector element (hM4Di). Then, the drug was accurately injected into the bilateral subthalamic nuclei through stereotactic surgery. After the surgery, combined with clozapine, the abnormal activity of the subthalamic nucleus was precisely intervened to improve the core motor symptoms of Parkinson's disease.
Conditions: Parkinson's Disease (PD)
Interventions: gene therapy
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